DelveInsight’s, “Charcot Marie Tooth Disease Pipeline Insight, 2025” report provides comprehensive insights about 10+ companies and 15+ pipeline drugs in Charcot Marie Tooth Disease pipeline landscape. It covers the Charcot Marie Tooth pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Charcot Marie Tooth therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
Curious about the latest updates in the Charcot Marie Tooth Pipeline? Click here to explore the therapies and trials making headlines @ Charcot Marie Tooth Pipeline Outlook Report
Key Takeaways from the Charcot Marie Tooth Pipeline Report
- On 23 October 2025, NMD Pharma A/S announced a Phase 2a study aims to evaluate the efficacy, safety and tolerability of NMD670 vs placebo administered twice a day (BID) for 21 days in ambulatory adult patients with Charcot-Marie-Tooth disease type 1 and type 2.
- DelveInsight’s Charcot Marie Tooth Pipeline report depicts a robust space with 10+ active players working to develop 15+ pipeline therapies for Charcot Marie Tooth treatment.
- The leading Charcot Marie Tooth Companies such as Pharnext SA, InFlectis BioScience, ENCell, HELIXMITH, Chong Kun Dang Pharmaceuticals, Addex Therapeutics, Augustine therapeutics, Novartis Pharmaceuticals, and others.
- Promising Charcot Marie Tooth Therapies such as PXT3003, NMD670, MD1003, Engensis (VM202), Remibrutinib, Teriflunomide, Cladribine (MAVENCLAD®) and others.
Want to know which companies are leading innovation in Charcot Marie Tooth? Dive into the full pipeline insights @ Charcot Marie Tooth Clinical Trials Assessment
Charcot Marie Tooth Overview
Charcot-Marie-Tooth disease (CMT) is a group of inherited neurological disorders that affect the peripheral nerves, which are responsible for movement and sensation in the limbs. Named after the three physicians who first described it in 1886, CMT is also known as hereditary motor and sensory neuropathy (HMSN). It is one of the most common inherited neurological disorders, affecting about 1 in 2,500 people. CMT is characterized by progressive muscle weakness and atrophy, primarily in the legs and feet, and, in later stages, in the hands and arms.
Charcot Marie Tooth Disease Emerging Drugs
- PXT3003: Pharnext SA
PXT3003, is a novel fixed-dose synergistic combination of baclofen, naltrexone and sorbitol formulated as an oral solution given twice a day. The three individual components of PXT3003 were selected to downregulate the overexpression of PMP22 protein, leading to improvement of neuronal signaling in dysfunctional peripheral nerves that are an essential part of the pathophysiology of this disease. PXT3003 could also have a positive effect on other cellular types of the motor unit such as the axon (direct protection), neuromuscular junctions or muscle cells. It is currently in Phase III stage of development for the treatment of Charcot Marie tooth disease type 1A.
- IFB-088: InFlectis BioScience
IFB-088 is an orally available small molecule capable of crossing the blood-brain barrier, a cell layer that protects the brain from disease-causing agents and toxins that may be present in the blood. IFB-088 also serves as an inhibitor of certain glutamate receptors. These receptors are involved in a process called excitotoxicity, in which neurons are over-stimulated and fire much more than needed, leading to nerve damage. IFB 088 has the potential to treat several demyelinating subtypes including CMT 1A, CMT 1B and CMT 1E, which affects 150,000 people in the U.S. and Europe. The Company is now planning a Phase II clinical trial in the United States and Europe. The drug is currently in Phase II stage of development for the treatment of Charcot Marie tooth disease.
If you’re tracking ongoing Charcot Marie Tooth Clinical trials, this press release is a must-read. Tap to see the breakthroughs @ Charcot Marie Tooth Treatment Drugs
Charcot Marie Tooth Companies
Pharnext SA, InFlectis BioScience, ENCell, HELIXMITH, Chong Kun Dang Pharmaceuticals, Addex Therapeutics, Augustine therapeutics, Novartis Pharmaceuticals, and others.
The Charcot Marie Tooth Pipeline report provides insights into:-
- The report provides detailed insights about companies that are developing therapies for the treatment of Charcot Marie Tooth with aggregate therapies developed by each company for the same.
- It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Charcot Marie Tooth Treatment.
- Charcot Marie Tooth Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
- Charcot Marie Tooth Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
- Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Charcot Marie Tooth market.
Charcot Marie Tooth Disease pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
- Intravenous
- Subcutaneous
- Oral
- Intramuscular
Charcot Marie Tooth Products have been categorized under various Molecule types such as
- Monoclonal antibody
- Small molecule
- Peptide
From emerging drug candidates to competitive intelligence, the Charcot Marie Tooth Pipeline Report covers it all – check it out now @ Charcot Marie Tooth Market Drivers and Barriers, and Future Perspectives
Scope of the Charcot Marie Tooth Pipeline Report
- Coverage- Global
- Charcot Marie Tooth Companies- Pharnext SA, InFlectis BioScience, ENCell, HELIXMITH, Chong Kun Dang Pharmaceuticals, Addex Therapeutics, Augustine therapeutics, Novartis Pharmaceuticals, and others.
- Charcot Marie Tooth Therapies- PXT3003, NMD670, MD1003, Engensis (VM202), Remibrutinib, Teriflunomide, Cladribine (MAVENCLAD®) and others.
- Charcot Marie Tooth Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
- Charcot Marie Tooth Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
Stay ahead in Healthcare Research – discover what’s next for the Charcot Marie Tooth Treatment landscape in this detailed analysis @ Charcot Marie Tooth Emerging Drugs and Major Players
Table of Content
- Introduction
- Executive Summary
- Charcot Marie Tooth Disease: Overview
- Pipeline Therapeutics
- Therapeutic Assessment
- Charcot Marie Tooth Disease– DelveInsight’s Analytical Perspective
- Late Stage Products (Phase III)
- PXT3003: Pharnext SA
- Drug profiles in the detailed report…..
- Mid Stage Products (Phase II)
- Drug Name: Company Name
- Drug profiles in the detailed report…..
- Early Stage Products (Phase I)
- IFB-088: InFlectis BioScience
- Drug profiles in the detailed report…..
- Preclinical and Discovery Stage Products
- Drug Name: Company Name
- Drug profiles in the detailed report…..
- Inactive Products
- Charcot Marie Tooth Disease Key Companies
- Charcot Marie Tooth Disease Key Products
- Charcot Marie Tooth Disease- Unmet Needs
- Charcot Marie Tooth Disease- Market Drivers and Barriers
- Charcot Marie Tooth Disease- Future Perspectives and Conclusion
- Charcot Marie Tooth Disease Analyst Views
- Charcot Marie Tooth Disease Key Companies
- Appendix
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